Intra-condensate demixing of TDP-43 inside stress granules generates pathological aggregates
Research output: Contribution to journal › Research article › Contributed › peer-review
Contributors
Abstract
Cytosolic aggregation of the nuclear protein TAR DNA-binding protein 43 (TDP-43) is associated with many neurodegenerative diseases, but the triggers for TDP-43 aggregation are still debated. Here, we demonstrate that TDP-43 aggregation requires a double event. One is up-concentration in stress granules beyond a threshold, and the other is oxidative stress. These two events collectively induce intra-condensate demixing, giving rise to a dynamic TDP-43-enriched phase within stress granules, which subsequently transition into pathological aggregates. Intra-condensate demixing of TDP-43 is observed in iPS-motor neurons, a disease mouse model, and patient samples. Mechanistically, intra-condensate demixing is triggered by local unfolding of the RRM1 domain for intermolecular disulfide bond formation and by increased hydrophobic patch interactions in the C-terminal domain. By engineering TDP-43 variants resistant to intra-condensate demixing, we successfully eliminate pathological TDP-43 aggregates in cells. We suggest that up-concentration inside condensates followed by intra-condensate demixing could be a general pathway for protein aggregation.
Details
| Original language | English |
|---|---|
| Pages (from-to) | 4123-4140.e18 |
| Journal | Cell |
| Volume | 188 |
| Issue number | 15 |
| Publication status | Published - 24 Jul 2025 |
| Peer-reviewed | Yes |
External IDs
| PubMedCentral | PMC12303766 |
|---|---|
| Scopus | 105005869906 |
| ORCID | /0000-0003-4017-6505/work/191040361 |
| ORCID | /0000-0003-0475-3790/work/191041724 |
| ORCID | /0000-0002-4281-7209/work/196680207 |
Keywords
Keywords
- Animals, DNA-Binding Proteins/metabolism, Disease Models, Animal, Humans, Mice, Motor Neurons/metabolism, Neurodegenerative Diseases/metabolism, Oxidative Stress, Protein Aggregates, Protein Aggregation, Pathological/metabolism, Protein Domains, Stress Granules/metabolism